First-line biologic therapy with tumor necrosis factor inhibitors for psoriatic arthritis: a prospective observational study

ABSTRACT BACKGROUND: Psoriatic arthritis (PsA) is a chronic inflammatory disease that affects multiple joints. It is associated with psoriasis and treated with synthetic and biologic drugs. OBJECTIVE: The objective of this study was to assess the outcomes of patients who received biologic therapy with tumor necrosis factor (TNF) inhibitors in terms of effectiveness, safety, functionality, and quality of life. DESIGN AND SETTING: A prospective observational study was performed at a single center in Belo Horizonte, Brazil. METHODS: Patients with PsA who received their first TNF inhibitor treatment were followed up for 12 months. Disease activity was measured using the Bath Ankylosing Spondylitis Disease Activity Index (BASDAI) and Clinical Disease Activity Index (CDAI). Functionality was measured using the Health Questionnaire Assessment (HAQ), and quality of life was evaluated using the European Quality of Life Five Dimensions (EQ-5D). Multiple linear regression was used to identify predictors of the clinical response at 12 months. RESULTS: A total of 143 patients treated with adalimumab or etanercept were evaluated. Most of the clinical measures were significantly improved at 12 months. However, 31%-51% of the patients did not achieve good clinical control. No differences were observed between adalimumab and etanercept, except for poor functionality at 12 months among patients treated with etanercept. The main predictors of a worse clinical response were female sex, etanercept use, poor functionality, or lower quality of life at baseline. The main adverse reactions were alopecia, headache, injection site reaction, sinusitis, flu, dyslipidemia, and infections. CONCLUSION: TNF inhibitor therapy was effective and safe. However, despite improvements in clinical measures, most patients did not achieve satisfactory control of the disease.

Diferencia Mínima Clínicamente Importante: Centrando la Toma de Decisiones en el Paciente / Clinically Important Minimal Difference: Centering Decision Making on the Patient

Introducción. Las condiciones de salud evaluadas en la práctica clínica o la investigación requieren ser interpretadas más allá de la significancia estadística. Por ello, el objetivo de este artículo especial es presentar elementos que permitan entender la diferencia mínima clínicamente importante (DCMI). La DCMI se define como la diferencia más pequeña en la puntuación en cualquier dominio o desenlace de interés que los pacientes son capaces de percibir como beneficiosa. Estudios que incorporan la percepción de los pacientes en la valoración de los hallazgos, permiten valorar de mejor manera la evidencia y con ello una mejor toma de decisiones. Es necesario seguir investigando e incorporando la DCMI, ya que, si bien en el último tiempo ha habido un avance en esta línea, aun es insuficiente en todos los ámbitos de la salud, la rehabilitación y la kinesiología.

Background. Health conditions evaluated in clinical practice or research need to be interpreted beyond statistical significance. Therefore, the objective of this special article is to present elements that allow us to understand the minimal clinically important difference (MCID). The MCID is defined as the smallest difference in score in any domain or outcome of interest that patients are able to perceive as beneficial. Studies that incorporate the perception of the patients in the evaluation of the findings, allow a better evaluation of the evidence and with it a better decision making. It is necessary to continue investigating and incorporating MCID, since, although there has been progress in this line in recent times, it is still insufficient in all areas of health, rehabilitation and kinesiology.

Self-management program (parqve) improves quality of life in severe knee osteoarthritis / Programa de autogestão (parqve) melhora a qualidade de vida na osteoartrite grave do joelho

ABSTRACT Objective: To evaluate the effects of the self-management program PARQVE in patients with severe knee osteoarthritis (KOA). Methods: Prospective randomized controlled clinical trial with 65 grade IV Kelgren & Lawrence (K&L) KOA patients who were allocated into groups: Control (CG) and Intervention (IG). Both groups received usual care. IG also participated in two days of multi-professional interventions about OA (causes and treatment) and received the program's DVD and book. Standing X-rays were obtained at inclusion and Ahlback's classification was registered. Western Ontario and McMaster Universities Index (WOMAC), Numerical Rating Scale (NRS), Lequesne, weight, and body mass index (BMI) were obtained at inclusion, and after 6, 12 and 24 months. Results: Groups were similar at baseline, despite higher WOMAC stiffness scores and a greater number of Ahlback's grade 4 and 5 in the IG. Only the IG improved WOMAC and total functions (p<0.001) during the study period above 12%, but did not reach the minimal clinically important difference of 20%. Best results were in one year. Non-significant improvements were observed without changes in body composition (P>0.05). Conclusions: Patients with severe KOA have mild to moderate function and quality of life improvement due to self-management program (PARQVE). Level of Evidence I; Therapeutic Studies; Prospective Randomized Controlled Trial.

RESUMO Objetivo: Avaliar os efeitos do programa de autocuidado PARQVE em pacientes com osteoartrite grave de joelho (OAJ). Métodos: Ensaio clínico prospectivo randomizado controlado com 65 pacientes Kelgren & Lawrence (K&L) grau IV que foram alocados nos grupos: Controle (GC) e Intervenção (GI). Ambos os grupos receberam cuidados habituais. O IG também participou de dois dias de intervenções multiprofissionais sobre OA (causas e tratamento) e seus membros receberam o DVD e o livro do programa. Raios-X em pé foram obtidos na inclusão e a classificação de Ahlback foi registrada. Western Ontario e McMaster Universities Index (WOMAC), Escala de classificação numérica (ECN), Lequesne, peso e índice de massa corporal (IMC) foram obtidos na inclusão, e aos 6, 12 e 24 meses. Resultados: Os grupos eram semelhantes no início do estudo, apesar de maiores escores de rigidez WOMAC e um número maior de pacientes de Ahlback grau 4 e 5 no GI. Apenas o GI melhorou em WOMAC e função total (p <0,001) acima de 12% durante o período de estudo. Os melhores resultados foram após um ano. Melhorias não significativas foram observadas na composição corporal (P> 0,05). Conclusões: Pacientes com OAJ grave apresentam melhora leve a moderada de função e qualidade de vida pelo programa de autogerenciamento (PARQVE). Nível de Evidência I; Estudos Terapêuticos; Estudo Clínico Prospectivo e Randomizado.

Establishing minimal clinically important differences of Quality of Life Instruments for Cancer Patients-Leukemia based on the distribution-based approach / 国际肿瘤学杂志

Objective:To develop the minimal clinically important difference (MCID) of Quality of Life Instruments for Cancer Patients-Leukemia (QLICP-LE) (V2.0).Methods:The quality of life of 101 patients with leukemia in First Affiliated Hospital of Kunming Medical University and First People′s Hospital of Yunnan Province from October 2011 to May 2012 were measured. The QLICP-LE (V2.0) was used for data collection, and the MCID for the overall score and scores of various domains of QLICP-LE (V2.0) were established by using the distribution-based approach including indexes of effect size, standard error of measurement (SEM), reliable change index, standardized response mean and responsiveness statistic, and the recommended values of MCID were determined through the consensus method.Results:The MCID formulated by the above five indexes were as follows: the total scale 1.4-9.3, physical functional domain 1.6-15.6, psychological functional domain 2.9-15.6, social functional domain 2.2-18.0, common symptoms and side-effects domain 1.7-17.1, common module 1.8-10.0, and the specific module 1.1-12.1. Through the expert consensus method, it was recommended to use the MCID results calculated by 1.96SEM: the total scale was 4, physical domain was 8, psychological domain was 8, social domain was 9, common symptoms and side-effects domain was 9, common module was 4, and the specific module was 6.Conclusion:Each index of distribution-based approach has its own advantages and disadvantages, which can be selected based on actual conditions. There is clinical significance when the score change of QLICP-LE (V2.0) of leukemia patients after treatment exceeds its MCID.

Improvements in hip osteoarthritis with lavage, triamcinolone and hylan g-f20 / Melhora na osteoartrite de quadril por lavagem, triancinolona e hilano

ABSTRACT Objective: To verify whether the use of Hylan G-F20 improves saline lavage and triamcinolone injection results in the treatment of hip osteoarthritis (HOA). Methods: 82 patients with HOA categorized as grades II and III severity, according to Kellgren and Lawrence criteria, were randomized into the groups: lavage and triamcinolone (G0); lavage, triamcinolone, and 2 mL of hylan G-F20 (G1); lavage, triamcinolone, and 4mL of hylan G-F20 (G2); lavage, triamcinolone, and 6mL of hylan G-F20 (G3). The VAS, range of motion (ROM), WOMAC, and Lequesne questionnaires were administered at baseline, one, three, six, and twelve months post-injection. Results: All groups showed clinically relevant improvements (> 20%) between baseline and first month post-injection, maintaining subjective results throughout the study period (p < 0.001). We found no differences between groups in any subjective evaluations (p > 0.05, for all). G2 and G3 obtained improved flexion results up to a year (p = 0.028). Hylan groups presented an improved external rotation since the first postoperative month and maintained the results up to a year (G1, p = 0.041; G2, p = 0.007), whereas G0 showed no improvement (p = 0.336). Conclusion: Hip lavage and triamcinolone injection, with or without the use of hylan, improves pain, function, and quality of life up to a year in HOA. Hylan may improve ROM up to one year. Level of Evidence IB, Randomized clinical trial.

RESUMO Objetivo: Verificar se o Hylan G-F20 melhora os resultados da lavagem e injeção de triancinolona na osteoartrite do quadril (OAQ). Métodos: 82 pacientes com HOA Kellgren e Lawrence graus II e III foram randomizados: lavagem e triamcinolona (G0); lavagem, triancinolona e 2 mL de Hylan G-F20 (G1); lavagem, triamcinolona e 4 mL de Hylan G-F20 (G2); lavagem, triancinolona e 6 mL de Hylan G-F20 (G3). A escala visual analógica (EVA), amplitude de movimento (ADM), questionários Womac e Lequesne foram obtidos no início, um, três, seis e doze meses após a injeção. Resultados: Todos os grupos apresentaram melhora clinicamente relevante (> 20%) entre o início e o primeiro mês, mantendo resultados subjetivos durante o estudo (p <0,001). Não foram encontradas diferenças entre os grupos nas avaliações subjetivas (p > 0,05). A flexão aumentou no G2 e G3 até um ano (p = 0,028). A rotação externa melhorou nos grupos Hylan no primeiro mês, mantendo os resultados até um ano (G1, p = 0,041; G2, p = 0,007), enquanto G0 nunca melhorou (p = 0,336). Conclusão: Lavagem do quadril e injeção de triancinolona, com ou sem Hylan, melhoram a dor, função e qualidade de vida até um ano na OAQ. Hylan pode melhorar a ADM até um ano. Nível de evidência IB, Ensaio clínico randomizado.

Functional assessment using short tests in a patient with pompe disease receiving enzyme replacement therapy: case report

ABSTRACT Introduction: Pompe disease is characterized by the deficiency of the acid alfa glucosidase enzyme, which leads to a glycogen accumulation mainly in cardiac and skeletal muscles. Its onset may be early or late; the late form is more difficult to handle given the variety of presentations. Enzyme replacement therapy has shown to improve gross motor function and lung function in patients. Case description: Female patient who presented chronic quadriparesis. She was diagnosed with Pompe disease, which required enzyme replacement therapy that helped improve the symptoms, which was evident with the performance of rapid functional evaluation tests. Discussion: Enzyme replacement therapy in Pompe disease modifies the natural history of the disease. A brief review of the literature about the functional tests that can be used to assess a patient with this disorder is presented. Conclusion: The 10-meter walk test, one-leg stance test, cervical flexion in supine position, five times sit to stand test, and coin rotation task are useful for clinical evaluation in patients with Pompe disease receiving enzyme replacement therapy.

RESUMEN Introducción. La enfermedad de Pompe se caracteriza por una alteración de la enzima alfa glucosidasa ácida lisosomal que produce acumulación de glicógeno, principalmente en el músculo esquelético y cardiaco. Se presentan formas de inicio temprano y tardío, esta última de más difícil manejo dada la múltiple variedad de presentaciones. La terapia de reemplazo enzimático ha demostrado mejorar la función motora gruesa y pulmonar de los pacientes con esta patología. Presentación del caso. Paciente femenino quien presentó cuadro crónico de cuadriparesia y a quien se le documentó enfermedad de Pompe, por lo que requirió terapia de reemplazo enzimático con mejoría franca de sintomatología, objetivizada con la realización de pruebas rápidas de evaluación funcional. Discusión. La terapia de reemplazo enzimático en la enfermedad de Pompe modifica la historia natural de la enfermedad. Se realiza una breve revisión de la literatura acerca de las pruebas funcionales que pueden ser utilizadas dentro de la evaluación del paciente con este trastorno. Conclusiones. La prueba de caminata de 10m, de equilibrio monopodal, de flexión cervical en supino, de levantarse-sentarse cinco veces y de la moneda resultan útiles para la evaluación clínica de los pacientes con enfermedad de Pompe en terapia de remplazo enzimático.

Minimal clinically important difference for the rhinoconjunctivitis quality of life questionnaire in allergic rhinitis in Thai population

BACKGROUND: Clinical measurement of the severity of allergic rhinitis (AR) can be assessed by symptoms score and patient quality of life (QoL). The magnitude of change in both symptoms and patient QoL should be considered in the management of AR. OBJECTIVE: The aim of this study was to determine the minimal clinically important difference (MCID) in AR in Thai population. METHODS: This prospective study recruited AR patients that attended our clinic during September 2011 to February 2012. The Rhinoconjunctivitis Quality of Life (Rcq-36) questionnaire was used to evaluate patient QoL. The Global Rating of Change Scale (GRCS) was used to assess improvement or deterioration in condition. The MCID was determined using an anchor-based method based on the GRCS, which was compared to the MCID determined by a distribution-based method based on the standard deviation (SD). RESULTS: Three hundred seventy-two patients with chronic rhinitis were recruited. Eighty-four of those had positive skin prick test and be diagnosed as AR. Of those, 79 completed the study and were included in the final analysis. A change of 2 points on the GRCS yielded an MCID for overall QoL of 0.21 ± 0.57. When applying the distribution-based method, an SD of 1.5 SD yielded an MCID for QoL of 0.27. For the rhinitis symptoms domain, the MCID was 0.42 ± 0.72. CONCLUSIONS: The MCID for overall QoL and rhinitis symptoms in Thai AR patients was found to be 0.21 ± 0.57 and 0.42 ± 0.72, respectively. MCID can be applied for determining the clinical significance of treatment efficacy in AR.

Acceptable Functional Outcomes and Patient Satisfaction Following Total Knee Arthroplasty in Asians with Severe Knee Stiffness: A Matched Analysis

BACKGROUND: This study evaluated outcomes following total knee arthroplasty for severely stiff knees in Asians. METHODS: Registry data of patients undergoing primary total knee arthroplasty between 2004 and 2013 were collected and retrospectively reviewed. Sociodemographic and anthropometric data together with the Oxford Knee Score and the Knee Society Score (Knee Society Knee Score and the Knee Society Function Score) were collected both preoperatively and postoperatively for up to 2 years. Case subjects consisted of patients with a preoperative flexion range of ≤ 20°. Control subjects consisted of patients with a preoperative flexion range of > 90°. Patients were matched for age, sex, and all preoperative scores in a 2:1 fashion. Two-year outcomes and 5-year revision rates were then compared between cases and controls. RESULTS: There were 28 cases and 56 controls. Cases had a significantly lower body mass index than the controls (p = 0.003) and had a longer hospital stay (p < 0.0001). At 2 years, cases had a significantly lower flexion range (p < 0.001) and a lower Knee Society Function Score (p = 0.020) than the controls. Cases had a significantly greater improvement in the flexion range (p < 0.001) postoperatively than controls. The mean change in functional outcomes at 2 years was comparable between the two groups. Seventy-one percent of the cases and 88% of controls were satisfied. There was a significant difference in the 5-year revision rate (10% vs 0%, p = 0.013). More cases were discharged to a rehabilitation facility compared to controls (p = 0.011). There were no differences in inpatient complication rates. CONCLUSIONS: Functional outcomes and patient satisfaction were acceptable following total knee arthroplasty in Asian patients with severe knee stiffness.

Single-Level Minimally Invasive Transforaminal Lumbar Interbody Fusion Provides Sustained Improvements in Clinical and Radiological Outcomes up to 5 Years Postoperatively in Patients with Neurogenic Symptoms Secondary to Spondylolisthesis

STUDY DESIGN: Retrospective review of prospective registry data. PURPOSE: To determine 5-year clinical and radiological outcomes of single-level instrumented minimally invasive transforaminal lumbar interbody fusion (MIS-TLIF) in patients with neurogenic symptoms secondary to spondylolisthesis. OVERVIEW OF LITERATURE: MIS-TLIF and open approaches have been shown to yield comparable outcomes. This is the first study to assess MIS-TLIF outcomes using the minimal clinically important difference (MCID) criterion. METHODS: The outcomes of 56 patients treated by a single surgeon, including the Oswestry disability index (ODI), neurogenic symptom score, short-form 36 questionnaire (SF-36), and visual analog scale (VAS) scores for back pain (BP), and leg pain (LP), were collected prospectively for up to 5 years postoperatively. Radiological outcomes included adjacent segment degeneration, fusion, cage subsidence, and screw loosening rates. RESULTS: Our patients were predominantly female (71.4%) and had a mean age of 53.7±11.3 years and mean body mass index of 25.7±3.7 kg/m². The mean operative time, blood loss, time to ambulation, and hospitalization were 167±49 minutes, 126±107 mL, 1.2±0.4 days, and 2.8±1.1 days, respectively. The mean fluoroscopic time was 58.4±33 seconds, and the mean postoperative intravenous morphine dose was 8±2 mg. Regarding outcomes, postoperative scores improved relative to preoperative scores, and this was sustained across various time points for up to 5 years (p<0.001). Improvements in ODI, SF-36, VAS-BP, and VAS-LP all met the MCID criterion. Notably, 5.4% of our patients developed clinically significant adjacent segment disease during follow-up, and 7 minor complications were reported. CONCLUSIONS: Single-level instrumented MIS-TLIF is suitable for patients with neurogenic symptoms secondary to lumbar spondylolisthesis and is associated with an acceptable complication rate. Both clinical and radiological outcomes were sustained up to 5 years postoperatively, with many patients achieving an MCID.

Diferencia mínima clínicamente importante en algunas escalasde medición utilizadas en enfermedades musculoesqueléticas / Minimal clinically important difference in some measurement scales used in musculoskeletal diseases

Objetivo: Presentar un enfoque práctico y útil de las escalas más usadas en el áreamusculoesquelética con su respectiva diferencia mínima clínicamente importante paraque los interesados puedan utilizar e interpretar correctamente los hallazgos de losestudios clínicos.Metodología: Revisión narrativa en donde a través de un consenso con especialistas dereumatología y ortopedia se seleccionaron las escalas que evaluaban función o dolor enhombro, codo, cadera y rodilla, y que tuvieran una puntuación metodológica ≥ 5/6 para serdescritas; además, se realizó una búsqueda en PubMed acerca del número de citaciones decada una de las escalas de medición evaluadas.Resultados: Fueron analizadas 23 escalas de funcionalidad y dolor: 6 de hombro, 4 de codo,3 de cadera, 4 de rodilla, 4 de cadera/rodilla y 2 generales, para las cuales se reportan lasrespectivas diferencias mínimas clínicamente importantes.Conclusiones: La diferencia mínima clínicamente importante es una herramienta útil quepermite a los clínicos en el área de enfermedades musculoesqueléticas utilizar e interpretarcorrectamente las escalas de medición y los efectos de las intervenciones medidas en losestudios clínicos...

Objective: To present a practical and useful approach to the most used scales in the musculoskeletal area with its own "Minimal Clinically Important Difference" (MCID) for those interested in using and interpreting the findings of clinical studies.Methods: A Narrative review, in which, by consensus with rheumatology and orthopedic specialists, a selection was made of the scales that evaluate function or pain in shoulder, elbow, hip and knee, and had a methodological score ≥ 5/6. , A PubMed search was also conducted to find the number of citations of each of the measurement scales evaluated.Results: A total of 23 scales of function and pain were analyzed: shoulder 6, elbow 4, hip 3, 4 knee, 4 hip/knee, and 2 general, in which the respective minimum clinically important difference was reported.Conclusions: The minimum clinically important differences is a useful tool for clinicians in the area of skeletal use and interpretation of measurement scales, and the effects of intervention measures in clinical studies on muscle diseases...